FDA de­rails Acor­da's mar­ket­ing pitch, cre­at­ing a cri­sis as flag­ship drug founders

Acor­da just en­tered a world of hurt.

With its patent pro­tec­tion crum­bling around its main­stay drug, the FDA has re­fused to file its ap­pli­ca­tion for CVT-301, the res­cue pro­gram that was des­ig­nat­ed to take the lead drug’s place. The RTF cre­ates an em­bar­rass­ing and per­haps crip­pling prob­lem for the biotech, which is fight­ing a last-ditch bat­tle to main­tain patent pro­tec­tion on its one big drug, Ampyra.

Ron Co­hen

Ac­cord­ing to Acor­da, the FDA says the com­pa­ny’s mar­ket­ing ap­pli­ca­tion, rushed to reg­u­la­tors as the com­pa­ny re­struc­tured and down­sized, is in­com­plete. Reg­u­la­tors said they were un­able to do a prop­er re­view as they don’t know what date Acor­da’s man­u­fac­tur­ing cen­ter will be ready for in­spec­tion and have a “ques­tion re­gard­ing the sub­mis­sion of the drug mas­ter pro­duc­tion record.”

Acor­da re­port­ed that the FDA al­so re­quest­ed ad­di­tion­al in­for­ma­tion at re­sub­mis­sion, which was not part of the ba­sis for the RTF.

Its shares quick­ly plunged 26% on the news.

Nor­mal­ly, a de­lay like this would be seen as a sticky prob­lem, but one that could po­ten­tial­ly be re­solved with­out huge de­lays. The prob­lem for Acor­da, though, is that the com­pa­ny has vir­tu­al­ly no lee­way for a sna­fu like this. Not on­ly does it leave Acor­da’s ex­ec­u­tive team with a black eye on ex­e­cu­tion, it cre­ates se­ri­ous doubts about its abil­i­ty to quick­ly and ef­fi­cient­ly line up a re­place­ment drug for its sales team by next sum­mer, as the lit­i­ga­tion over its patents for Ampyra comes to a head.

Leerink’s Paul Mat­teis found the whole thing puz­zling and dis­con­cert­ing, con­sid­er­ing the like­li­hood of a loom­ing ap­proval. And he still has ques­tions about what is go­ing on at Acor­da.

It seems like the best case sce­nario would be a re-sub­mis­sion around the end of this year, and its en­cour­ag­ing that FDA did not make any re­quests re­gard­ing the ef­fi­ca­cy or safe­ty da­ta. Nonethe­less, even af­ter speak­ing with the com­pa­ny, the RTF is mys­te­ri­ous to us, and we ex­pect the stock to be un­der pres­sure un­til opac­i­ty is lift­ed.

As I re­port­ed in June, while re­view­ing the late-stage da­ta sub­mit­ted to the FDA on the in­haled lev­odopa, any set­back on CVT-301 would be a cat­a­stro­phe for Acor­da.

Acor­da shift­ed to sur­vival mode in ear­ly April af­ter a US dis­trict court tossed four key patents on its flag­ship drug Ampyra, leav­ing it with one to stand on in­to next year. The com­pa­ny chopped 20% of its staff as it scram­bles to re­struc­ture while gam­bling that it can field new drugs in short or­der.

Now Acor­da is left to des­per­ate­ly seek a quick meet­ing with reg­u­la­tors to clear up any of these ques­tions. But the FDA is not known for scram­bling a re­sponse in any of these sit­u­a­tions, leav­ing Acor­da’s time­line in the air just when it most needs cer­tain­ty. Just weeks ago one of its biggest in­vestors pushed CEO Ron Co­hen to auc­tion the com­pa­ny off rather than risk the con­se­quences of fail­ing at its high wire act. The com­pa­ny pushed back, but a sale may come back on the ta­ble now whether or not it likes the idea.

“We will work with the FDA as quick­ly as pos­si­ble to ad­dress the open is­sues and to clar­i­fy the path to suc­cess­ful­ly re-file our ap­pli­ca­tion,” said Co­hen in a state­ment. “We re­main con­fi­dent in IN­BRI­JA’s da­ta pack­age and its promise as an im­por­tant new ther­a­py for peo­ple with Parkin­son’s dis­ease. We see tremen­dous long-term val­ue in its sol­id clin­i­cal pro­file, sig­nif­i­cant com­mer­cial op­por­tu­ni­ty and strong IP, and we re­main fo­cused on work­ing to bring pa­tients this im­por­tant new ther­a­py.”

Eli Lilly CEO David Ricks at the Rose Garden, May 26, 2020 (Evan Vucci/AP Images)

Eli Lil­ly lines up a block­buster deal for Covid-19 an­ti­body, right af­ter it failed a NI­AID tri­al

Two days after Eli Lilly conceded that its antibody bamlanivimab was a flop in hospitalized patients, the US government is preparing to make it a blockbuster.

The pharma giant reported early Wednesday that it struck a deal to supply the feds with 300,000 vials of the drug at a cost of $375 million — once it gets an EUA stamp from the FDA. And once that 2-month supply deal is done, the government has an option on another 650,000 doses on the same terms — which could potentially add another $800 million.

Patrick Soon-Shiong at the JP Morgan Healthcare Conference, Jan. 13, 2020 (David Paul Morris/Bloomberg via Getty Images)

Af­ter falling be­hind the lead­ers, dissed by some ex­perts, biotech show­man Patrick Soon-Sh­iong fi­nal­ly gets his Covid-19 vac­cine ready for a tri­al. But can it live up to the hype?

In January, when dozens of scientists rushed to start making a vaccine for the then-novel coronavirus, they were joined by an unlikely compatriot: Patrick Soon-Shiong, the billionaire doctor most famous for making big, controversial promises on cancer research.

Soon-Shiong had spent the last 4 years on his “Cancer Moonshot,” but part of his project meant buying a small Seattle biotech that specialized in making common-cold vectors, called adenoviruses, to train the immune system. The billionaire had been using those vectors for oncology, but the company had also developed vaccine candidates for H1N1, Lassa fever and other viruses. When the outbreak began, he pivoted.

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Jude Samulski, Marianne De Backer

Bay­er buys a biotech ‘race horse’ with a $4B deal — $2B in cash — aimed at go­ing big in­to gene ther­a­py

In the latest sign that Big Pharma wants a leading place in the push to develop a new generation of cell and gene therapies, Bayer is stepping up today with a $2 billion cash deal to buy out one of the fast-moving pioneers in the field, while adding up to $2 billion more in milestones if the new pharma subsidiary can deliver the goods.

As part of a continuing series of deals engineered by Bayer BD chief Marianne De Backer, the pharma player has snapped up Asklepios, more commonly referred to in more casual fashion as AskBio. And they are paying top dollar for a Research Triangle Park-based company that raised $225 million a little more than a year ago to back the brainchild of Jude Samulski, the gene therapy pioneer out of the University of North Carolina Gene Therapy Center.

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En­her­tu picks up an­oth­er win for As­traZeneca and Dai­ichi Sankyo, join­ing the pri­or­i­ty re­view lane for gas­tric can­cer

Five months after Enhertu received twin breakthrough therapy designations, AstraZeneca and Daiichi Sankyo are one step closer to nabbing another approval for their potential blockbuster drug.

The companies announced Wednesday morning that their billion-dollar antibody-drug conjugate has received priority review for HER2 positive metastatic gastric cancer. Already approved in the US for third-line metastatic breast cancer patients that are HER2 positive, Enhertu’s gastric cancer PDUFA date is scheduled for the first quarter of 2021.

Sci­en­tists warn Amer­i­cans are ex­pect­ing too much from a coro­n­avirus vac­cine

The White House and many Americans have pinned their hopes for defeating the Covid-19 pandemic on a vaccine being developed at “warp speed.” But some scientific experts warn they’re all expecting too much, too soon.

“Everyone thinks COVID-19 will go away with a vaccine,” said William Haseltine, chair and president of Access Health International, a foundation that advocates for affordable care.

No­var­tis CEO Vas Narasimhan signs off on a $231M deal to try some­thing new in the R&D fight against SARS-CoV-2

Patrick Amstutz was baptized by pandemic fire early on.

He and colleagues attended the notorious Cowen conference in early March that included some of the top Biogen execs who helped trigger a superspreader event in Boston. Heading back to his post as CEO of Molecular Partners in Switzerland, the outbreak was sweeping through Italy, triggering near panic in some quarters and creeping into the voices of people he knew, including one friend on the Italian side of the country.

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Cedric Francois, Apellis CEO (Optum via YouTube)

UP­DAT­ED: So­bi bets $250M cash, about $1B in mile­stones for rights to a C3 ther­a­py be­ing pushed through 5 piv­otal tri­als

A couple years after licensing Novimmune’s emapalumab and turning around a quick FDA OK, Stockholm-based Sobi is betting up to $1.2 billion for rights to another rare disease drug.

The company is shelling out $250 million upfront and adding up to $915 million in milestones for rights to develop and commercialize Apellis Pharmaceuticals’ drug pegcetacoplan outside the US. Together, the companies will see the systemic C3 therapy through five registrational trials in hematology, nephrology and neurology.

Christian Rommel (via Roche)

Bay­er fol­lows R&D deal spree by raid­ing Roche's can­cer group for its new re­search chief

The day after Bayer signed off on a $4 billion deal designed to put the company among the leaders in gene therapy development, the pharma giant has recruited a new chief for its R&D division. And they opted for an expert in the cancer field.

Christian Rommel, Roche’s head of discovery and early-stage oncology development, has been tapped to take over the job. Joerg Moeller, who got the top research post after early- and late-stage development roles were combined 2 years ago, is hitting the exit “to pursue other career opportunities.”

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Once re­ject­ed, Kala's dry eye drug now gains en­try to a field where No­var­tis is groom­ing its own block­buster

When the FDA slapped a rejection on Kala Pharma’s dry eye drug last August, the biotech’s execs promised investors that a third Phase III study — they had already started at that point — would reverse their fortune.

Today they made good on that promise, clinching an approval for Eysuvis, an ocular corticosteroid being positioned as a first-line, short term treatment of dry eye disease.

Boasting a technology invented by Bob Langer out of MIT, Eysuvis is a corticosteroid, loteprednol etabonate, delivered by mucus-penetrating particles. It promises to enhance penetration into target tissue on the ocular surface, achieving an effect quicker than systemic corticosteroids and stronger than over-the-counter eye drops.